Drug Target Review

MAY 7, 2024

Why are biomarkers particularly critical in the context of rare diseases? Developing novel therapeutic approaches for rare diseases poses inherent challenges due to the limited populations available for testing. How do AI-enhanced approaches accelerate the drug development process for rare diseases?

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SCIENMAG: Medicine & Health

MARCH 5, 2024

WHAT: A weekly injection of semaglutide was safe and reduced the amount of fat in the liver by 31% in people with HIV and metabolic dysfunction-associated steatotic liver disease (MASLD), according to a presentation today at the 2024 Conference on Retroviruses and Opportunistic Infections (CROI) in Denver.

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The Pharma Data

OCTOBER 28, 2021

Bayer will present new renal and cardiovascular (CV) analyses from the comprehensive finerenone (Kerendia®) clinical trial program, including the Phase III FIGARO-DKD and FIDELIO-DKD studies, and the prespecified pooled analysis FIDELITY at the American Society of Nephrology (ASN)’s Kidney Week 2021 from 4-7 November.

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DrugBank

JULY 20, 2023

Rare diseases, therefore, present compelling opportunities for Drug Development. Since each disease is present in small groups of patients, this research is also fraught with complex challenges. We're excited to expand our data offering by launching a new Rare Disease add-on to complement our Clinical Trial dataset.

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Drug Target Review

NOVEMBER 29, 2023

Our primary focus is to design and develop RNA therapies for liver diseases. Using humans as the model, we use an approach called deep phenotyping to explore the relationships between cells, genes, biological pathways and patterns of disease. At present, we test discarded human livers deemed not good enough for transplant.

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PPD

NOVEMBER 7, 2023

Food and Drug Administration (FDA) draft guidance, “Diversity Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Populations in Clinical Trials.” The issue of including diverse populations that represent disease prevalence and country and regional demographics is not new to industry.

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The Pharma Data

NOVEMBER 9, 2021

– More than 30 Abstracts Across HDV, HCV, HBV, NASH and PSC Reflect Gilead’s Ongoing Commitment to Addressing Challenges Facing People Living With Liver Diseases –. Gilead Lores,Inc.

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PPD

OCTOBER 9, 2023

“Patient-centric” is used frequently to describe today’s clinical trial and health care landscape. The industry applauds that point of view and strives to include a patient-centric approach in all aspects of clinical trial designs. Education on the disease state and regular reinforcement.

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Vial

JUNE 18, 2024

The success of clinical trials hinges on increasing access to participation by all eligible patients, including populations that have been underrepresented due to the barriers highlighted below. Representation, Underrepresentation & Diversity in Clinical Trials Studies indicate a need to increase patient accessibility to clinical trials.

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Conversations in Drug Development Trends

APRIL 5, 2024

For others, this rare day is aptly the day we honor rare disease communities. Observed annually on the last day of February since 2008, Rare Disease Day has grown into a global movement for raising awareness, promoting research, and advocating for improved access to rare disease treatments and support services.

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Vial

JUNE 24, 2024

Contract research organizations (CROs) and pharmaceutical companies can leverage these cutting-edge technologies to streamline clinical trials and introduce automation in drug discovery. As clinical trials grow in complexity, the volume of data being gathered and utilized for these studies is expanding.

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The Pharma Data

AUGUST 30, 2021

Positive high-level results from the FoCus Phase III trial in Wilson disease showed ALXN1840 met the primary endpoint with a statistically significant improvement in daily mean copper mobilisation from tissues, demonstrating superiority compared with standard-of-care (SoC) treatments. Wilson disease. 1, 2, 3.

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Broad Institute

OCTOBER 23, 2023

By Makenzie Kohler October 23, 2023 People of South Asian ancestry around the world have more than double the risk of developing cardiometabolic diseases like diabetes, heart attack, and stroke compared to other populations.

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PPD

MAY 18, 2023

For neurodegenerative disease research, the care partner, or caregiver, is equally important as the person with dementia. Even with stronger awareness of studies, drug developers must contend with the inherent lack of trust between the dementia community and clinical trials. There’s little time to lose.

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Conversations in Drug Development Trends

JULY 8, 2024

By: Lisle Kingery, PhD, Therapeutic Strategy Lead, Neuroscience With depression rates at an all-time high and limited new treatments available, depression clinical trials are more important than ever. Treatment Treatment can take various forms, including therapy, medication, a combination of both, or a clinical trial.

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The Pharma Data

MAY 31, 2022

the completion of enrollment of its Phase 1 (Ph1), open-label trial of pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson’s disease (PD). Ph1 clinical trial is to evaluate the safety, tolerability, and?preliminary?efficacy?of of BRT-DA01 in patients with Parkinson’s disease.?. preliminary?efficacy?of

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The Pharma Data

NOVEMBER 14, 2020

(Nasdaq: GILD) and Novo Nordisk A/S (NASDAQ Copenhagen: NOVO B) today announced results from a Phase 2 proof-of-concept trial. The results were presented at The Liver Meeting Digital Experience (TLMdX), November 13–16, 2020 (Late Breaker #L02). Across all groups, 5–14% of people discontinued any trial treatment due to AEs.

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Drug Target Review

JUNE 3, 2024

How does COUR Pharmaceuticals’ immune-modifying nanoparticle platform differ from traditional approaches to treating immune-mediated diseases? By encapsulating specific self-antigens within our biodegradable nanoparticles, we enable the targeted delivery of the CNPs to antigen-presenting cells (APCs) primarily in the spleen and liver.

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The Pharma Data

MAY 22, 2023

DUPIXENT® (DUPILUMAB) LATE-BREAKING PHASE 3 COPD RESULTS PRESENTED AT ATS AND SIMULTANEOUSLY PUBLISHED IN THE NEW ENGLAND JOURNAL OF MEDICINE Regeneron Pharmaceuticals, Inc. The Dupixent Phase 3 results were presented in the 2023 American Thoracic Society (ATS) International Conference session “New England Journal of Medicine and JAMA.

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The Pharma Data

MARCH 16, 2022

The Independent Data Monitoring Committee recommended that the EMPA-KIDNEY trial be stopped early, following a formal interim assessment EMPA-KIDNEY is the largest and broadest dedicated SGLT2 inhibitor trial in chronic kidney disease to date Detailed results are expected to be presented later this year.

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biobide

FEBRUARY 2, 2024

Pediatric cancers exhibit differences at the genetic level compared to the same form of adult disease, which may influence the selected treatment. Some pediatric cancers, such as leukemias, present a high survival percentage with the available treatment regimens but cause systemic toxicities, and a subset of patients eventually relapse.

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The Pharma Data

JULY 21, 2021

Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company focused on the development and commercialization of prophylactic vaccines for infectious diseases with significant unmet medical need, and Pfizer Inc. If successful, this trial could enable the inclusion of a pediatric population in the Phase 3 trial.

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The Pharma Data

MAY 26, 2023

Pfizer Presents Scientific Advancements from its Leading Oncology Portfolio at ASCO 2023 Annual Meeting will present data across its Oncology portfolio and growing pipeline, covering multiple tumor types and novel mechanisms of action at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago from June 2 through June 6.

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PPD

MARCH 21, 2024

When participating in clinical trials, pediatric populations require distinct considerations for their safety and well-being — which can present unique challenges for drug developers. Five Strategies to Tailor Your Pediatric Clinical Trials to the Child’s Needs 1.

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The Pharma Data

MARCH 13, 2021

Further, prespecified exploratory analyses showed donanemab slowed the accumulation of tau across key brain regions in patients affected by Alzheimer’s disease. “This is the first late-stage study in Alzheimer’s disease to meet its primary endpoint at the primary analysis. Apostolova, M.D.,

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The Pharma Data

JUNE 7, 2023

The research presented reinforces the efficacy of LUMAKRAS ® /LUMYKRAS ® (sotorasib) in advanced non-small cell lung cancer (NSCLC) and metastatic colorectal cancer (mCRC). This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology.

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The Premier Consulting Blog

OCTOBER 11, 2022

Clinical trials for ultra-rare diseases can be particularly challenging to mount due to small, geographically-dispersed patient populations. For such trials, the US Food and Drug Administration (FDA) may allow the use of credible real-world data (RWD) and real-world evidence (RWE) in lieu of data collected in a Phase 3 trial.

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Advarra

JUNE 13, 2024

Recombinant DNA technologies and genetically modified biological agents are being adapted for a wide scope of therapeutic applications, and their use is becoming increasingly common in clinical trials. For example, a site conducting only protocols involving mRNA may be able to leverage a generic SOP covering any mRNA protocol at that site.

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Vial

FEBRUARY 21, 2024

There is a strong demand for innovative products that are essential in monitoring, preventing, and treating diseases or disabilities. Clinical trials are needed to determine whether a medical device can produce sufficient proof of its efficacy. This is where contract research organizations (CROs) come into play.

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The Pharma Data

AUGUST 30, 2021

New Kisqali ® (ribociclib)* overall survival (OS) results from MONALEESA-2 trial in HR+/HER2? Health-related quality of life, pain and safety outcomes from phase III VISION trial of investigational radioligand therapy 177 Lu-PSMA-617 in patients with metastatic castration-resistant prostate cancer. 19, 2:10 PM CEST].

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The Pharma Data

JULY 29, 2021

Eisai and Biogen Present Preliminary Assessment of the Clinical Effects of Lecanemab Following 18 Months of Treatment in the Open-Label Extension of the Phase 2 Proof of Concept Study at 2021 Alzheimer’s Association International Conference (AAIC) . United States and virtually from July 26 to 30, 2021 (Presentation No.:

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The Pharma Data

MARCH 11, 2021

Eli Lilly and Company (NYSE: LLY) today announced that data from programs across its oncology portfolio and pipeline will be presented at the 2021 American Association for Cancer Research (AACR) Annual Meeting, to be held virtually April 10-15, 2021. LY3484356, an oral SERD, is currently being studied in a Phase 1/2 clinical trial.

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The Pharma Data

APRIL 11, 2023

The presentation highlights the synergistic anti-cancer effects of a PSMA (prostate-specific membrane antigen)-actinium-225 conjugate in combination with darolutamide in preclinical prostate cancer models. Data from all three areas of scientific focus will be showcased during this year’s meeting.

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The Pharma Data

SEPTEMBER 6, 2021

First data to be presented from the phase II coopERA Breast Cancer study evaluating neoadjuvant giredestrant treatment for oestrogen receptor (ER)-positive, HER2-negative breast cancer. Breast Cancer Highlights. Lung Cancer Highlights.

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The Pharma Data

APRIL 12, 2022

20% variant allele frequency reduction – At the time of analysis with > 2 year follow up the survival estimate was 100% in patients who had improvements in bone marrow fibrosis or variant allele frequency – Results were presented at the American Association for Cancer Research annual meeting.

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The Pharma Data

MAY 16, 2022

AbbVie (NYSE: ABBV) today announced that 27 abstracts across its gastroenterology portfolio will be presented at the Digestive Disease Week (DDW) Annual Meeting, May 21-24, 2022, in San Diego and virtually. “We are proud to present data on a range of digestive conditions across our portfolio and pipeline. .

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PPD

OCTOBER 17, 2024

There has also been an increase in government and regulatory support for CGT trials, in conjunction with an increase in investments for these products to get to market. Site selection presents barriers and opportunities As cell therapies expand in Asia-Pacific, areas with large hospitals are often the main hubs for innovation.

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